Journal ArticleDOI
Design and development of polymers for gene delivery
TLDR
With the growing understanding of polymer gene-delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based gene-Delivery systems will become an important tool for human gene therapy.Abstract:
The lack of safe and efficient gene-delivery methods is a limiting obstacle to human gene therapy. Synthetic gene-delivery agents, although safer than recombinant viruses, generally do not possess the required efficacy. In recent years, a variety of effective polymers have been designed specifically for gene delivery, and much has been learned about their structure–function relationships. With the growing understanding of polymer gene-delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based gene-delivery systems will become an important tool for human gene therapy.read more
Citations
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Journal ArticleDOI
Non-viral vectors for gene-based therapy
Hao Yin,Rosemary Lynn Kanasty,Ahmed A. Eltoukhy,Arturo J. Vegas,J. Robert Dorkin,Daniel G. Anderson +5 more
TL;DR: The biological barriers to gene delivery in vivo are introduced and recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems are discussed, some of which are currently undergoing testing in clinical trials.
Journal ArticleDOI
Polymer conjugates as anticancer nanomedicines
TL;DR: There is growing optimism that ever more sophisticated polymer-based vectors will be a signficant addition to the armoury currently used for cancer therapy.
Journal ArticleDOI
Mucus-penetrating nanoparticles for drug and gene delivery to mucosal tissues.
TL;DR: The tenacious mucus barrier properties that have precluded the efficient penetration of therapeutic particles are described and the design and development of new mucus-penetrating particles that may avoid rapid mucus clearance mechanisms are reviewed to provide targeted or sustained drug delivery for localized therapies in mucosal tissues.
Journal ArticleDOI
Polymers for Drug Delivery Systems
TL;DR: The origins and applications of stimuli-responsive polymer systems and polymer therapeutics such as polymer-protein and polymer-drug conjugates are reviewed and the latest developments in polymers capable of molecular recognition or directing intracellular delivery are surveyed to illustrate areas of research advancing the frontiers of drug delivery.
Journal ArticleDOI
Bioconjugated quantum dots for in vivo molecular and cellular imaging
TL;DR: The synthesis and development of state-of-the-art QD probes and their use for molecular and cellular imaging are discussed and key issues for in vivo imaging and therapy, such as nanoparticle biodistribution, pharmacokinetics, and toxicology are examined.
References
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Journal ArticleDOI
A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine
Otmane Boussif,Frank Lezoualc'h,Maria Antonietta Zanta,Mojgan Mergny,Daniel Scherman,Barbara A. Demeneix,Jean-Paul Behr +6 more
TL;DR: Together, these properties make PEI a promising vector for gene therapy and an outstanding core for the design of more sophisticated devices because its efficiency relies on extensive lysosome buffering that protects DNA from nuclease degradation, and consequent lysOSomal swelling and rupture that provide an escape mechanism for the PEI/DNA particles.
Journal ArticleDOI
Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure
Philip L. Felgner,Thomas R. Gadek,Marilyn Holm,Richard Bolton Roman,Hardy W. Chan,Michael Wenz,Jeffrey P. Northrop,Gordon M. Ringold,Mark Danielsen +8 more
TL;DR: Depending upon the cell line, lipofection is from 5- to greater than 100-fold more effective than either the calcium phosphate or the DEAE-dextran transfection technique.
Journal ArticleDOI
Starburst Dendrimers: Molecular-Level Control of Size, Shape, Surface Chemistry, Topology, and Flexibility from Atoms to Macroscopic Matter
TL;DR: Starburst dendrimers are three-dimensional, highly ordered oligomeric and polymeric compounds formed by reiterative reaction sequences starting from smaller molecules—“initiator cores” such as ammonia or pentaerythritol.
Journal ArticleDOI
Gene therapy -- promises, problems and prospects
Inder M. Verma,Nikunj V. Somia +1 more
TL;DR: The prospects are good — by the year 2010, gene therapy may be as routine a practice as heart transplants are today.